Successful Development Strategies for Repurposed Drugs to Accelerate Drug Development in Rare Diseases

<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d15953072e85">Drug repurposing offers a cost-effective and time-efficient strategy to bring therapies to patients with rare diseases. However, the development path until product approval is complex for repurposed drugs, with challenges around meeting regulatory requirements as well as data and market exclusivities. This work explores the key elements for successful regulatory strategies of repurposed drugs in rare disease indications. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d15953072e87">A systematic review was conducted using EMA’s database for medicinal products to identify repurposed drugs that have completed the marketing authorization procedure in the EU as of today. For each drug, the available approval documentation was systematically reviewed to derive best practices and strategies on how to address EMA’s authorization requirements for repurposed drugs. Thiswork illustrates these key elements through a series of case studies. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d15953072e89">In total, 30 repurposed drugs for rare disease were found in EMA’s database out of which approximately two third of the applications were authorized and approximately one third of the applications were withdrawn. Aligning the chosen regulatory approval pathway with product-specific data requirements in terms of quality, nonclinical, clinical data determines the success or failure of the application. Effective regulatory interactions during development were identified as one of the key elements for success. Additional regulatory guidance and incentives are needed within the EU development framework to promote the repurposing of drugs in rare diseases.